ANAVEX2-73 (blarcamesine) Receives Compassionate Use Authorization for Pediatric Patients With Rett Syndrome

Anavex Life Sciences Corp. recently announced the first patients with Rett syndrome from the ANAVEX2-73-RS-003 study (EXCELLENCE) are able to continue treatment with ANAVEX2-73 (blarcamesine) due to Health Canada’s approval of the Special Access Program (SAP), allowing for compassionate use following completion of the 48-week Open-Label Extension study.

Special Access Program (SAP) applications are granted at a physicians’ request on behalf of patients. Based on the safety profile of ANAVEX2-73 (blarcamesine), as well as clinical evidence that the study drug will continue to benefit patients, Anavex will continue its responsibility to support patients post study by facilitating the supply of ANAVEX2-73 (blarcamesine) to patients in Canada. Approved compassionate programs are also ongoing in the UK and Australia, representing all regions of the Rett Syndrome study participation.

“Today there is no specific treatment for Rett syndrome available in Canada and the Ontario Rett Syndrome Association (O.R.S.A.) is pleased to see Health Canada has just granted several investigators’ requests to access for this study drug that may be beneficial to their patients,” said Sabrina Millson, President of the Ontario Rett Syndrome Association.

Health Canada’s Special Access Program (SAP) for drugs enables drugs that are not marketed in Canada to be requested by practitioners for the treatment, diagnosis, or prevention of serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or are unavailable either as marketed products, or through enrollment in clinical trials.

Christopher U Missling, PhD, President and Chief Executive Officer of Anavex, said “While our clinical studies with ANAVEX2-73 (blarcamesine) are very critical to collect clinical information for regulatory purposes, we also recognize the long-term commitment to our investigators, patients and families. Therefore, post clinical studies, we are keen to provide supply to the investigators that have requested compassionate use of ANAVEX2-73 (blarcamesine) for their patients on a global basis. In compliance with local authorities, many patients from our clinical studies are now able to continue treatment with ANAVEX2-73 (blarcamesine).”

Anavex Life Sciences Corp. is a publicly traded biopharmaceutical company dedicated to the development of novel therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders, including Alzheimer’s disease, Parkinson’s disease, Rett syndrome, and other central nervous system (CNS) diseases, pain, and various types of cancer. Anavex’s lead drug candidate, ANAVEX2-73 (blarcamesine), has successfully completed a Phase 2a and recently a Phase 2b/3 clinical trial for Alzheimer’s disease, a Phase 2 proof-of-concept study in Parkinson’s disease dementia, and both a Phase 2 and a Phase 3 study in adult patients with Rett syndrome. ANAVEX2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective, and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX2-73 for the treatment of Parkinson’s disease. ANAVEX3-71, which targets sigma-1 and M1 muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid, and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. For more information, visit www.anavex.com.